People in France with severe hemophilia A who switched to Elocta (efmoroctocog alfa) — an extended half-life replacement therapy — had fewer infusions and a reduced rate of joint bleeds, a real-world study reports. Notably, those who switched to Elocta had a higher bleeding rate, including joint bleeds,…

Nearly one-third of people with hemophilia B have antibodies against a subtype of an adeno-associated virus (AAV) — called AAV6 — that is used as an experimental gene therapy carrier, a U.K. study found. Because such antibodies could render the treatment that the carrier holds ineffective, these patients would…

Most women of childbearing age have normal monthly periods. For women like me with bleeding disorders, monthly periods are a “shark week event” with horrific bleeding and pain. The pain made me sweat, shake, and vomit. The severe bleeding had other consequences: I was iron-deficient, anemic, and exhausted. After work,…

I watch my son put pencil to paper and cannot help noticing the change in his face. The tension from just a few minutes ago gives way to peace. The minute he draws the first line on the page, he’s transported into a world that offers him rest and spiritual…

When my kids were little, people reminded me to enjoy my children because they would be in college before I knew it. I never thought much about that sentiment. With both of my sons living with severe hemophilia, I felt like I was going from one thing to the…

Treatment with an antibody designed to specifically block the anti-blood clotting, or anticoagulant, activity of an enzyme called activated protein C (APC) significantly reduced bleeding and related mortality in hemophilia A and hemophilia B mouse models, a study shows. The antibody, called HAPC1573, was also found to improve…

To those caring for a spouse or partner with chronic illness: Maybe you knew about your partner’s condition from the get-go. Or maybe they developed a chronic illness or disability somewhere along the road, and you stuck around, ever the loyal companion. You may feel bound by spousal duties…

European authorities have recommended conditional marketing authorization for Roctavian (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s one-time gene therapy for adults with severe hemophilia A. This positive decision, from the Committee for Medicinal Products for Human Use (CHMP), makes Roctavian the first gene therapy for hemophilia A to be recommended for European…

Dear Newly Diagnosed Sister, Welcome! You are now part of a supportive community full of love, courage, and inspirational superheroes. Always remember that you are not alone. I was diagnosed with von Willebrand disease and hemophilia B in 1975, at age 10. Your diagnosis Diagnosis of…

An innovative gel containing two snake venom proteins known to help blood clotting, rapidly and effectively forms stable clots and reduces bleeding even in the presence of a blood-clotting disorder, as is the case in hemophilia, a preclinical study shows. The gel, which transitions to a liquid form below…